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Enrolling by Invitation

NCT Number: NCT07413224

Qualitative Effects of AEF0217 in Down Syndrome People

Following the AEF0217-102 trial (NCT05748405), which investigated the effects of AEF0217 versus placebo, participants with Down syndrome and their caregivers who took part in the trial are invited to share their perceptions of what they experienced or observed, in an exploratory manner.

This qualitative study collects information through a specific questionnaire developed for this purpose.

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Key information

Conditions

Down Syndrome (Trisomy 21) Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Down Syndrome Genetic Diseases, Inborn Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital del Mar Research Institute

Barcelona, 08003, Spain

About this study

This is a non-interventional, follow-up, cross-sectional, mixed-methods (qualitative and quantitative) study.

Participants and their caregivers who will be enrolled are those who have received either AEF0217 or placebo and who have completed the previous trial.

The participant and their cargiver will be contacted prospectively to assess their interest in participating in the study.

The participant and their caregiver will sign an electronic informed consent form and will receive a link to a questionnaire developed using EU Survey to be completed.

Data will be collected at a single time point but may refer retrospectively to past experiences related to the previous trial. Data from all participants and caregivers who complete the questionnaire will be included in the analysis.

Given the observational and descriptive nature of the study, the quantitative analysis will be primarily descriptive.

The study will be conducted in accordance with the ethical principles derived from the Declaration of Helsinki (75th General Assembly, Helsinki, Finland, October 2024).

In addition, the study will be conducted in compliance with the protocol, Regulation (EU) 2016/679 (General Data Protection Regulation, GDPR), and the regulatory requirements applicable at the study site.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants having completed the trial AEF0217-102
  • Being the caregiver of the participant
  • Provided written informed consent (or assent in case of individuals with Down syndrome with no capacity to legally sign the consent)

Exclusion criteria

1- Not wanting to provide a written informed consent

Treatment and study plan

Primary outcomes

  1. - Overall, how would the caregiver describe the experience with the effect of the treatment the participant received during the trial?

    Time frame: Day 1

    • Rating of the global perception of the treatment period (Likert scale). Scale in 5 points (Very positive, Somewhat positive, Neutral, Somewhat negative, Very negative).
    • Completed by the caregiver

Other outcomes

  1. Overall, how the study satisfied the effects the caregiver was expecting in the participant after receiving the treatment??

    Time frame: Day 1

    • Rating of the global perception of the treatment period (Likert scale). Scale in 5 points (Very positive, Somewhat positive, Neutral, Somewhat negative, Very negative)
    • Completed by the caregiver

  2. Which treatment does the caregiver believe the participant received before knowing the actual assignment (active drug or placebo)

    Time frame: Day 1

    • Rating Scale in 3 points (AEF0217, Placebo, I am not sure)
    • completed by the caregiver

  3. Was the caregiver's assumption about the treatment allocation correct?

    Time frame: Day 1

    • Rating Scale in 2 points Yes or No
    • Completed by the caregiver

  4. If the caregiver noticed an improvement what type of changes did the caregiver observe after the end of the treatment period?

    Time frame: Day 1

    Rating as follows:

    • The improvement continued up to this day
    • The improvement continued for some time and then started to decline
    • The improvement remained stable up to this day
    • The improvement remained stable for some time and then started to decline
    • The improvement started to decline shortly after the end of the treatment
    • After the decline the function returned to and stabilized at the level prior to the study
    • After the decline the functioning becomes progressively worse than the level prior to the study.

    Percentage calculated.

    • Completed by the caregiver

  5. Ability to manage personal needs: eating, dressing, bathing, hygiene

    Time frame: Enrolment day

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  6. Ability to carry out household responsibilities, such as cleaning up, completing chores, and preparing food

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  7. Improvement in his/her engagement in structured activities or as a student, an apprentice or in his/her paid job

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  8. Improvement in his/her ability to adapt to new situations

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  9. Improvement in his/her ability to handle responsibilities

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  10. Improvement in his/her ability to face challenges and stress

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  11. Improvement in his/her ability to apply numerical concepts in everyday situations, such as understanding time, dates, and handling of money (cash)

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  12. Improvement his/her ability to navigate life outside the home, as for example have an improved safety awareness and use of transportation

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  13. Ability to relate and interact with others

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  14. Ability to maintain and develop relationships

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  15. He/she is more empathic

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  16. Increased activity in participating in recreational activities with others

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  17. Improvement in adapting to new various social situations

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  18. Ability to take part in a conversation/communication

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  19. He/she listens more attentively and/or responds more appropriately

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  20. Understand better spoken and nonverbal information

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  21. Can better express needs, thoughts, and emotions using verbal language

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  22. Write more often and/or use better grammar and/or spelling

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  23. Reads and better understand texts.

    Time frame: Day 1

    • Amplitude of the observed changes
    • Rating: numeric score from 0 to 10 with "0" = No improvement at all and "10" = Very marked improvement
    • Completed by the caregiver

  24. Thinking about the participant's abilities while taking the treatment, what changes (if any) did the caregiver notice?

    Time frame: Day 1

    • Qualitative (open text)
    • Thematic analysis to identify perceived domains of change and salient themes
    • Completed by the caregiver

  25. In the caregiver's opinion, what impact did the changes, if any, had on the participant's wellbeing and the day-to-day activities of the family?

    Time frame: Day 1

    • Qualitative (open text)
    • Qualitative coding will be performed independently by two researchers using NVivo 15 (or similar). Discrepancies will be resolved through consensus discussions. Themes will be validated through iterative review and triangulated with quantitative findings to ensure coherence.
    • Thematic analysis to identify perceived domains of change and salient themes
    • Completed by the caregiver

  26. In the caregiver's opinion, what impact did the changes, if any, have on the participant's interaction with others in the social ?

    Time frame: Day 1

    • Qualitative (open text)
    • Thematic analysis to identify perceived domains of change and salient themes
    • Completed by the caregiver

  27. Does the participant remember the effects of the treatment with AEF0217?

    Time frame: Day 1

    • Binary variable: Yes or no
    • Completed by the participant with Down Syndrome

  28. Did the participant notice any change to the better in abilities or behaviors after participating in the AEF0217 study?

    Time frame: Day 1

    • Only if the question 28 is answered "Yes"
    • Rated using a 4 levels scale (Big changes, Some changes, No change, Worsening)
    • Completed by the participant with Down Syndrome

  29. If the participant observed changes, in which areas did they happen ?

    Time frame: Day 1

    • Only if the question 29 is answered "big changes" or some changes"
    • Rated using a multiple-choice question (more than one answer is possible)
    • My abilities to understand
    • My ability to speak
    • My ability to write
    • My ability to concentrate
    • My ability to pay attention
    • My ability to control my behavior
    • My abilities to learn at school
    • My ability to take care of myself
    • My ability to interact with others
    • My ability to perform my daily abilities
    • My ability to execute my job
    • Completed by the participant with Down Syndrome

  30. Does the participant think the treatment helped daily life ?

    Time frame: Day 1

    • Only if the question 29 is answered "big changes" or some changes"
    • Rated using a 4 levels scale (Yes, a lot/Yes, a little/Not really/I don't know)
    • Completed by the participant with Down Syndrome

Sponsors and collaborators

Lead sponsor

Aelis Farma

Industry

Collaborators

  • Hospital del Mar Research Institute (IMIM)

Registry information

Official study title

Qualitative Evaluation of Perceived Effects Elicited by AEF0217 in Individuals With Down Syndrome

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Feb 17, 2026
Registry last updated
Apr 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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